Value in Health最新文献_第6页

Exploring Heterogeneity in the Cost-Effectiveness of High-Flow Nasal Cannula Therapy in Acutely Ill Children—Insights From the Step-Up First-line Support for Assistance in Breathing in Children Trial Using a Machine Learning Method 探索急性病儿童高流量鼻插管 (HFNC) 治疗的成本效益异质性--利用机器学习方法从 FIRST-ABC 高级试验中获得的启示。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2025-01-01 DOI: 10.1016/j.jval.2024.08.008

Zaid Hattab MS , Silvia Moler-Zapata PhD , Edel Doherty PhD , Zia Sadique PhD , Padmanabhan Ramnarayan MD , Stephen O’Neill PhD

Objectives

To investigate heterogeneity in the cost-effectiveness of high-flow nasal cannula (HFNC) therapy compared with continuous positive airway pressure (CPAP) for acutely ill children requiring noninvasive respiratory support.

Methods

Using data from the First-line Support for Assistance in Breathing in Children trial, we explore heterogeneity at the patient and subgroup levels using 2 causal forest approaches and a seemingly unrelated regression approach for comparison. First-line Support for Assistance in Breathing in Children is a noninferiority randomized controlled trial (ISRCTN60048867) involving 24 UK pediatric intensive care units. The Step-up trial focuses on acutely ill children aged 0 to 15 years, requiring noninvasive respiratory support. A total of 600 children were randomly assigned to HFNC and CPAP groups in a 1:1 allocation ratio, with 94 patients excluded because of data unavailability.

Results

The primary outcome is the incremental net monetary benefit (INB) of HFNC compared with CPAP, using a willingness-to-pay threshold of £20 000 per quality-adjusted life year gain. INB is derived from total costs and quality-adjusted life years at 6 months. Subgroup analysis showed that some subgroups, such as male children, those aged less than 12 months, and those without severe respiratory distress at randomization, had more favorable INB results. Patient-level analysis revealed heterogeneity in INB estimates, particularly driven by the cost component, with greater uncertainty for those with higher INBs.

Conclusions

The estimated overall INB of HFNC is significantly larger for specific patient subgroups, suggesting that the cost-effectiveness of HFNC can be heterogeneous, which highlights the importance of considering patient characteristics in evaluating the cost-effectiveness of HFNC.

目的研究在需要无创呼吸支持的急性期患儿中，高流量鼻插管（HFNC）疗法与持续气道正压（CPAP）疗法成本效益的异质性：利用儿童呼吸辅助系统第一线支持（FIRST-ABC）试验的数据，我们使用两种因果森林方法和一种看似不相关的回归（SUR）方法在患者和亚组水平上探讨了异质性。FIRST-ABC 是一项非劣效性随机对照试验（ISRCTN60048867），涉及 24 个英国儿科重症监护病房。该 "升级 "试验主要针对需要无创呼吸支持的 0 至 15 岁急症儿童。共有 600 名儿童按 1:1 的分配比例被随机分配到 HFNC 组和 CPAP 组，其中 94 名患者因无法获得数据而被排除：主要结果是 HFNC 与 CPAP 相比的增量净货币收益 (INB)，使用的支付意愿阈值为每 QALY 收益 20,000 英镑。INB 由总成本和 6 个月的质量调整生命年 (QALY) 得出。亚组分析表明，一些亚组，如男性儿童、年龄小于 12 个月的儿童和随机时无严重呼吸困难的儿童，INB 结果更有利。患者层面的分析显示，INB估计值存在异质性，尤其是在成本部分，INB较高者的不确定性更大：结论：对于特定的患者亚群，HFNC 的总体 INB 估计值要大得多，这表明 HFNC 的成本效益可能存在异质性，突出了在评估 HFNC 成本效益时考虑患者特征的重要性。

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引用次数: 0

Challenges in Value Assessment for One-Time Gene Therapies for Inherited Retinal Diseases: Are We Turning a Blind Eye? 一次性基因疗法治疗遗传性视网膜疾病的价值评估挑战：我们是否视而不见？

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2025-01-01 DOI: 10.1016/j.jval.2024.08.009

Jake Hitch MRes , Tom Denee MScBA , Simon Brassel MSc , Jennifer Lee MBA , Michel Michaelides MD, FRCOphth , Jacob Petersen MSc , Sarah Alulis MPH , Lotte Steuten PhD

Objectives

X-linked retinitis pigmentosa (XLRP) is a rare inherited retinal disease with no available treatment. Gene therapies in clinical trials will pose challenges for health technology assessment (HTA) if found to be safe and effective. We evaluated 2 of these challenges, namely acceptability and difficulties in assessing value beyond short-term patient health and healthcare savings and discounting in economic evaluation.

Methods

We conducted a narrative literature review on the socioeconomic burden of XLRP to identify relevant components of value for a hypothetical gene therapy from a societal perspective and to assess their relative importance. We compared the resulting value profile against the value frameworks of three European HTA agencies. We also reviewed their guidelines on discounting and potential discounting issues specific to XLRP.

Results

Much of the societal value of an XLRP gene therapy is likely to originate from productivity effects, carer spillovers, and value elements related to patient uncertainty. The evidence on these effects, however, is often limited, making it difficult for HTA agencies to assess them. Cost-effectiveness results are likely to be highly sensitive to the discount rate, and discounting will compound the effects of omitting important sources of value.

Conclusions

We have identified and detailed important components of societal value, key evidence gaps, and potential discounting issues for an XLRP gene therapy, which can inform future value assessments. Many of these may apply to gene therapies in other disease areas. Revisiting existing HTA approaches is recommended to ensure these are fit for purpose for such new classes of treatment.

目的：X 连锁色素性视网膜炎（XLRP）是一种罕见的遗传性视网膜疾病，目前尚无治疗方法。临床试验中的基因疗法如果安全有效，将给 HTA 带来挑战。我们研究了其中的两个挑战，即在评估短期患者健康和医疗节省以外的价值时的可接受性和困难，以及经济评估中的贴现：方法：我们对 XLRP 的社会经济负担进行了叙述性文献综述，以从社会角度确定假设基因疗法的相关价值组成部分，并评估其相对重要性。我们将得出的价值概况与欧洲三家 HTA 机构的价值框架进行了比较。我们还审查了它们的折现准则以及 XLRP 特有的潜在折现问题：XLRP基因疗法的大部分社会价值可能来自于生产力效应、护理人员的溢出效应以及与患者不确定性相关的价值要素。然而，有关这些效应的证据往往很有限，因此 HTA 机构很难对其进行评估。成本效益结果很可能对贴现率高度敏感，而贴现将加剧忽略重要价值来源的影响：我们为 XLRP 基因疗法确定并详细说明了社会价值的重要组成部分、关键证据差距以及潜在的贴现问题，这些都可以为未来的价值评估提供参考。其中许多可能适用于其他疾病领域的基因疗法。建议重新审视现有的 HTA 方法，以确保这些方法适用于此类新的治疗类别。

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引用次数: 0

Analytical Methods for Comparing Uncontrolled Trials With External Controls From Real-World Data: A Systematic Literature Review and Comparison With European Regulatory and Health Technology Assessment Practice 从真实世界数据中比较无对照试验与外部对照的分析方法：系统性文献综述及与欧洲监管和健康技术评估实践的比较。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2025-01-01 DOI: 10.1016/j.jval.2024.08.002

Milou A. Hogervorst PharmD, PhD , Kanaka V. Soman MSc , Helga Gardarsdottir PharmD, PhD , Wim G. Goettsch PhD , Lourens T. Bloem PharmD, PhD

Objectives

This study aimed to provide an overview of analytical methods in scientific literature for comparing uncontrolled medicine trials with external controls from individual patient data real-world data (IPD-RWD) and to compare these methods with recommendations made in guidelines from European regulatory and health technology assessment (HTA) organizations and with their evaluations described in assessment reports.

Methods

A systematic literature review (until March 1, 2023) in PubMed and Connected Papers was performed to identify analytical methods for comparing uncontrolled trials with external controls from IPD-RWD. These methods were compared descriptively with methods recommended in method guidelines and encountered in assessment reports of the European Medicines Agency (2015-2020) and 4 European HTA organizations (2015-2023).

Results

Thirty-four identified scientific articles described analytical methods for comparing uncontrolled trial data with IPD-RWD–based external controls. The various methods covered controlling for confounding and/or dependent censoring, correction for missing data, and analytical comparative modeling methods. Seven guidelines also focused on research design, RWD quality, and transparency aspects, and 4 of those recommended analytical methods for comparisons with IPD-RWD. The methods discussed in regulatory (n = 15) and HTA (n = 35) assessment reports were often based on aggregate data and lacked transparency owing to the few details provided.

Conclusions

Literature and guidelines suggest a methodological approach to comparing uncontrolled trials with external controls from IPD-RWD similar to target trial emulation, using state-of-the-art methods. External controls supporting regulatory and HTA decision making were rarely in line with this approach. Twelve recommendations are proposed to improve the quality and acceptability of these methods.

目的：概述科学文献中利用患者个人真实世界数据（IPD-RWD）将无对照药物试验与外部对照进行比较的分析方法。此外，还将这些方法与欧洲监管机构和卫生技术评估（HTA）机构在指南中提出的建议及其在评估报告中描述的评价进行比较：方法：在PubMed和Connected Papers上进行了一次系统的文献综述（截至2023年3月1日），以确定将IPD-RWD中的非对照试验与外部对照进行比较的分析方法。将这些方法与方法指南中推荐的方法以及欧洲药品管理局（2015-2020 年）和四个欧洲 HTA 组织（2015-2023 年）的评估报告中出现的方法进行了描述性比较：34 篇已确定的科学文章介绍了将非对照试验数据与基于 IPD-RWD 的外部对照进行比较的分析方法。各种方法涵盖了混杂控制和/或从属删减、缺失数据校正以及分析比较建模方法。七份指南还侧重于研究设计、RWD 质量和透明度方面，其中四份指南推荐了与 IPD-RWD 进行比较的分析方法。监管报告（n=15）和 HTA 评估报告（n=35）中讨论的方法通常基于综合数据，并且由于提供的细节较少而缺乏透明度：文献和指南建议采用最先进的方法，将非对照试验与IPD-RWD的外部对照进行比较，类似于目标试验模拟。支持监管和 HTA 决策的外部对照很少符合这种方法。为提高这些方法的质量和可接受性，我们提出了 12 项建议。

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引用次数: 0

Economic Evaluation of Fasting Mimicking Diet Versus Standard Care in Diabetic Patients on Dual or Triple Medications at Baseline in the United States: A Cost-Utility Analysis 对美国基线服用双重或三重药物的糖尿病患者进行空腹模拟饮食与标准护理的经济评估：成本效用分析。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2025-01-01 DOI: 10.1016/j.jval.2024.08.003

Dany Habka PhD , William C. Hsu MD , Joseph Antoun PhD

Objectives

According to most guidelines, dietary interventions are essential in the management of diabetes. Fasting has emerged as potential therapeutic regimes for diabetes. The proof-of-concept study and the fasting in diabetes treatment trial are the first to explore the clinical impact of the Fasting Mimicking Diet (FMD) in patients with type 2 diabetes mellitus. Their results showed that FMD cycles improve glycemic management and can be integrated into usual care complementary to current guidelines. This economic evaluation aims to assess the 10-year quality-of-life effects, cost implications, and cost-effectiveness of adding a 3-year FMD program to diabetes standard care in diabetic population on dual or triple medications at baseline from the perspective of the US payer.

Methods

We constructed a microsimulation model in TreeAge using a published US-specific diabetes model. The model was populated using FMD effectiveness outcomes and publicly available clinical and economic data associated with diabetes complications, use of diabetes medications, hypoglycemia incidence, direct medical costs in 2021 USD, quality of life, and mortality. All benefits were discounted by 3%.

Results

This cost-utility analysis showed that the FMD program was associated with 11.4% less diabetes complications, 67.2% less overall diabetes medication use, and 45.0% less hypoglycemia events over the 10-year simulation period. The program generated an additional effectiveness benefit of 0.211 quality-adjusted life year and net monetary benefit of 41 613 USD per simulated patient. Thus, the FMD program is cost saving.

Conclusions

These results indicate that the FMD program is a beneficial first-line strategy in T2DM management.

目的：根据大多数指南，饮食干预对糖尿病的治疗至关重要。禁食已成为糖尿病的潜在治疗方案。概念验证研究和禁食治疗糖尿病试验首次探讨了禁食模拟饮食（FMD™）对 T2DM 患者的临床影响。他们的研究结果表明，FMD™ 循环可改善血糖管理，并可作为现行指南的补充纳入常规护理中。本经济评估旨在从美国支付方的角度，评估在糖尿病标准治疗的基础上增加为期 3 年的 FMD™ 计划对糖尿病患者 10 年生活质量的影响、成本影响以及成本效益：我们使用已发布的美国糖尿病模型在 TreeAge 中构建了一个微观模拟模型。该模型使用 FMD™ 效果、与糖尿病并发症相关的公开临床和经济数据、糖尿病药物使用、低血糖发生率、2021 美元的直接医疗成本、生活质量和死亡率。所有收益均按 3% 的折扣计算：成本效用分析表明，在 10 年模拟期内，FMD™ 计划可使糖尿病并发症减少 11.4%，糖尿病药物总用量减少 67.2%，低血糖事件减少 45.0%。该计划为每位模拟患者带来了 0.211 QALY 的额外有效性收益和 41,613 美元的净货币收益。因此，FMD™ 计划可以节约成本：这些结果表明，FMD™ 计划是治疗 T2DM 的一线策略。

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引用次数: 0

A Review and Comparative Case Study Analysis of Real-World Evidence in European Regulatory and Health Technology Assessment Decision Making for Oncology Medicines 欧洲肿瘤药物监管和 HTA 决策中真实世界证据的回顾和比较案例研究分析。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2025-01-01 DOI: 10.1016/j.jval.2024.09.007

Jihong Zong MD, PhD, MS , Adina Rojubally PhD , Xiaoyun Pan PhD , Birgit Wolf MSc , Scott Greenfeder PhD , Alexander Upton MSc , Joette Gdovin Bergeson PhD, MPA

Objectives

Real-world evidence (RWE) is valuable in supporting regulatory and health technology assessment (HTA) decisions; however, the actual contribution to approvals remains elusive. This study aimed to review RWE approaches and use in oncology medicine approvals in Europe and understand cohesion and discrepancy in the acceptance of the RWE by the European Medicines Agency (EMA) and European HTA bodies.

Methods

This scoping review involved a search of the EMA database, National Institute for Health and Care Excellence (NICE), Gemeinsamer Bundesausschuss (G-BA), and Haute Autorité de Santé (HAS) websites to identify final reports and appraisals for oncology medicines with references to RWE. The selection was guided by research terms associated with RWE study designs, data sources, and outcomes. Qualitative analysis was used to systemize the data. Case studies assessed by more than one agency were selected for comparative assessment of RWE approach, use, and acceptability.

Results

RWE was mainly leveraged as an external control for indirect treatment comparisons or contextualization to support clinical trial results by the EMA, NICE, G-BA, and HAS. However, this approach was mostly rejected due to methodology biases. Comparative assessment of RWE acceptability for the same oncology medicines across agencies suggests discrepancies between EMA and European HTA bodies and among NICE, G-BA, and HAS.

Conclusions

There is diverging acceptance of RWE in EMA and European HTA bodies with no clear consensus on the most effective way to leverage RWE in approvals. With the introduction of the joint European Union Joint Clinical Assessment in 2025, it is crucial for European HTA bodies and EMA to develop synergetic standards for the use of RWE to ensure equitable and timely access to medicines.

目标：真实世界证据（RWE）在支持监管和 HTA 决策方面很有价值，但其在审批中的实际贡献仍难以捉摸。本研究旨在回顾 RWE 在欧洲肿瘤药物审批中的方法和使用情况，了解 EMA 和欧洲 HTA 机构在接受 RWE 方面的一致性和差异：本次范围界定审查涉及对 EMA 数据库、NICE、G-BA 和 HAS 网站的搜索，以确定提及 RWE 的肿瘤药物最终报告和评价。在选择时使用了与RWE研究设计、数据来源和结果相关的研究术语。定性分析用于系统化数据。为了对 RWE 方法、使用和可接受性进行比较评估，选择了由一个以上机构评估的案例研究：结果：RWE 主要被 EMA、NICE、G-BA 和 HAS 用作外部对照，用于间接治疗比较或背景分析，以支持临床试验结果。然而，由于方法上的偏差，这种方法大多遭到拒绝。各机构对相同肿瘤药物的 RWE 可接受性的比较评估表明，EMA 和欧洲 HTA 机构之间以及 NICE、G-BA 和 HAS 之间存在差异：结论：EMA 和欧洲 HTA 机构对 RWE 的接受程度存在差异，对于在审批中利用 RWE 的最有效方法尚未达成明确共识。随着 2025 年欧盟联合临床评估的引入，欧洲 HTA 机构和 EMA 必须为使用 RWE 制定协同标准，以确保公平、及时地获得药品。

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引用次数: 0

Deriving a Preference-Weighted Measure for People With Hypoglycemia From the Hypo-RESOLVE QoL 从 Hypo-RESOLVE-QoL 中得出低血糖患者的偏好加权测量值。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2025-01-01 DOI: 10.1016/j.jval.2024.10.3800

Donna Rowen PhD , Emily McDool PhD , Jill Carlton PhD , Philip Powell PhD , Richard Norman PhD

Objectives

Hypoglycemia affects the health-related quality of life (HRQoL) of people living with diabetes (PwD), and existing preference-weighted measures do not capture all important aspects. The study aimed to generate a preference-weighted measure capturing the HRQoL impact of hypoglycemia in PwD.

Methods

Items for the health-state classification system were selected from the hypoglycemia-specific Hypo-RESOLVE QoL measure using relevance in cognitive interviews, translatability, suitability for valuation, endorsement by patient advisors and experts, and psychometric performance in a large survey of PwD. Second, an online valuation survey using discrete choice experiment (DCE) with survival attribute was conducted with members of the UK public. DCE data were modeled using conditional logit analysis and results scaled to produce preference weights for the classification system on a scale in which 1 is equivalent to full health, 0 is equivalent to dead, and below 0 is worse than dead.

Results

The health-state classification system consists of 8 items reflecting the factors of the Hypo-RESOLVE QoL (psychological, social, and physical aspects). The valuation survey was completed by 1000 members of the UK public, representative for age and sex. Good understanding of DCE tasks was demonstrated. The item “do what I want to do in my life” had the largest preference weight, and “find it hard to stop thinking about my glucose levels” had the smallest.

Conclusions

This study generated Hypo-RESOLVE QoL-8D, a preference-weighted measure capturing the HRQoL impact of hypoglycemia in PwD, with UK general public preference weights. The measure can be generated from Hypo-RESOLVE QoL data.

目的：低血糖会影响糖尿病患者（PwD）的健康相关生活质量（HRQoL），而现有的偏好加权测量方法并不能捕捉到所有重要方面。本研究旨在制定一种偏好加权测量方法，以捕捉低血糖对糖尿病患者 HRQoL 的影响：健康状态分类系统的项目是从低血糖症专用的 Hypo-RESOLVE QoL 测量中筛选出来的，筛选时考虑了以下因素：认知访谈中的相关性、可翻译性、评估的适用性、患者顾问和专家的认可度，以及大型残疾人调查中的心理测量表现。其次，对英国公众进行了一项在线估价调查，采用的是带有生存属性的离散选择实验（DCE）。利用条件对数分析法对离散选择实验数据进行建模，并对结果进行缩放，以产生分类系统的偏好权重，其中 1 表示完全健康，0 表示死亡，低于 0 表示比死亡更糟：健康状况分类系统由 8 个项目组成，反映了 Hypo-RESOLVE QoL 的各因素（心理、社会和生理方面）。估值调查由 1000 名英国公众完成，他们的年龄和性别均具有代表性。他们对 DCE 任务有很好的理解。其中，"做我一生中想做的事 "的偏好权重最大，而 "发现很难停止思考我的血糖水平 "的偏好权重最小：本研究利用英国普通公众的偏好权重，生成了低血糖影响残疾人 HRQoL 的偏好权重测量指标 Hypo-RESOLVE QoL-8D。该指标可从 Hypo-RESOLVE QoL 数据中生成。

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引用次数: 0

Evolving Concept of Value in Health Economics and Outcomes Research: Emerging Tools for Innovation and Access to Cell and Gene Therapies for Rare Diseases. 健康经济学和结果研究中不断发展的价值概念：罕见疾病细胞和基因疗法的创新和获取的新兴工具。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-12-30 DOI: 10.1016/j.jval.2024.12.006

Walter Toro, Min Yang, Anish Patel, Su Zhang, Omar Dabbous, Louis P Garrison

Objectives: Recent scientific breakthroughs have propelled the development of disease-modifying and potentially curative cell and gene therapies (CGTs) for rare diseases, including those diseases previously considered untreatable. The unique characteristics of CGTs, however, pose challenges to the traditional methods of therapy value determination, reimbursement, and outcome evaluation used by regulatory and assessment agencies for product approval and market access. Notably, CGTs are one-time or short-course treatments, often first-in-class (precluding direct comparisons with effective alternatives), and have health benefits that are largely realized over time.

Methods: We summarized emerging health economics and outcomes research (HEOR) solutions in 5 areas pertaining to CGTs for rare diseases using examples from literature and recently approved therapies. These solutions include the collection of real-world data to generate real-world evidence, patient centricity and novel value assessment frameworks, evolution of economic evaluation methodologies, novel reimbursement models and management of affordability, and health equity and societal benefits.

Results: Advances in HEOR methods have informed the design and collection of long-term real-world data for CGT efficacy extrapolation, use of novel (including surrogate) endpoints, integration of the patient's voice and preferences, and application of sophisticated statistical methodology and artificial intelligence/machine learning techniques.

Conclusions: Continuing innovation in HEOR is expected to contribute to improved health outcomes, reduced costs, and enhanced access, ultimately enabling more efficient delivery of CGTs to patients living with rare diseases.

目的：最近的科学突破推动了罕见病（包括以前认为无法治疗的疾病）的疾病修饰和潜在治愈的细胞和基因疗法（cgt）的发展。然而，cgt的独特特性给监管和评估机构用于产品审批和市场准入的传统治疗价值确定、报销和结果评估方法带来了挑战。值得注意的是，cgt是一次性或短期治疗，通常是一流的（排除了与有效替代方案的直接比较），并且随着时间的推移，其健康益处在很大程度上得以实现。方法：我们利用文献中的例子和最近批准的治疗方法，总结了与罕见病cgt相关的五个领域的新兴健康经济和结果研究（HEOR）解决方案。这些解决方案包括收集真实世界数据（RWD）以产生真实世界证据（RWE）、以患者为中心和新的价值评估框架、经济评估方法的发展、新的报销模式和可负担性管理，以及卫生公平和社会效益。结果：HEOR方法的进步为CGT疗效推断的长期RWD的设计和收集提供了信息，使用了新的（包括替代的）终点，整合了患者的声音和偏好，并应用了复杂的统计方法和人工智能/机器学习技术。结论：HEOR的持续创新预计将有助于改善健康结果、降低成本和提高可及性，最终使罕见病患者能够更有效地获得cgt。

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引用次数: 0

An Exploratory Study of Alternative Time Frames and Descriptors for EQ-5D-5L in Obstructive Airway Diseases Using Mixed Methods. 使用混合方法对EQ-5D-5L在阻塞性气道疾病中的替代时间框架和描述符进行探索性研究

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-12-28 DOI: 10.1016/j.jval.2024.12.001

引用次数: 0

The Psychometric Properties of the EQ-HWB and EQ-HWB-S in Patients With Breast Cancer: A Comparative Analysis With EQ-5D-5L, FACT-8D, and SWEMWBS. EQ-HWB和EQ-HWB- s在乳腺癌患者中的心理测量特性：与EQ-5D-5L、FACT-8D和SWEMWBS的比较分析

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-12-27 DOI: 10.1016/j.jval.2024.12.003

Stevanus Pangestu, Fredrick Dermawan Purba, Hari Setyowibowo, Yohana Azhar, Clara Mukuria, Fanni Rencz

Objectives: The EQ Health and Wellbeing (EQ-HWB) is a new generic measure that captures constructs beyond health-related quality of life, with a 25-item long form and a shorter 9-item version (EQ-HWB-S). This study aimed to assess the psychometric performance of both versions in breast cancer, which is the most prevalent cancer worldwide, and compare them with other instruments.

Methods: A longitudinal survey in Indonesia (2023-2024) with 300 female patients used the EQ-HWB, 5-level EQ-5D (EQ-5D-5L), Functional Assessment of Cancer Therapy - General (from which Functional Assessment of Cancer Therapy Eight Dimension [FACT-8D] was derived), and Warwick-Edinburgh Mental Wellbeing Scale (WEMWBS, from which the Short WEMWBS was derived). Distributional characteristics, convergent validity, known-group validity (Student's t test or analysis of variance), test-retest reliability, and responsiveness were assessed.

Results: All patients reported problems in at least 1 EQ-HWB item. The EQ-HWB-S index (11%) had a lower ceiling than the EQ-5D-5L (35%) and the Short WEMWBS (15.3%), but not the FACT-8D (5%). EQ-HWB-S index values correlated strongly with EQ-5D-5L (r = 0.73) and FACT-8D index values (r = 0.70), whereas EQ-HWB level sum scores correlated strongly with Functional Assessment of Cancer Therapy - General (r = 0.69) and moderately with WEMWBS (r = 0.49). The EQ-HWB and EQ-HWB-S discriminated across known groups comparably with the EQ-5D-5L and FACT-8D with large effect sizes according to EuroQol visual analog scale groups, number of symptoms, and general health and exhibited excellent instrument-level test-retest reliability (intraclass correlations, 0.79-0.83) and acceptable responsiveness (standardized response means, |0.24| to |0.97|).

Conclusions: This study represents one of the first validations of the EQ-HWB and EQ-HWB-S in any clinical population. Both instrument versions demonstrate robust psychometric performance. The EQ-HWB-S can be recommended to inform resource allocation decisions of breast cancer treatments.

目的：情商健康与幸福是一种新的通用测量，它捕获了健康相关生活质量以外的结构，有一个25项的长表格（EQ- hwb）和一个较短的9项版本（EQ- hwb - s）。本研究旨在评估这两种版本在全球最常见的乳腺癌中的心理测量性能，并将其与其他工具进行比较。方法：在印度尼西亚（2023-24）对300名女性患者进行纵向调查，使用EQ-HWB， EQ-5D-5L，癌症治疗功能评估（FACT-G， FACT-8D来源于此），沃里克-爱丁堡心理健康量表（WEMWBS， SWEMWBS来源于此）。评估了分布特征、收敛效度、已知组效度（学生t检验或方差分析）、重测信度和反应性。结果：所有患者都报告了至少一个EQ-HWB项目的问题。EQ-HWB-S指数（11%）的上限低于EQ-5D-5L（35%）和SWEMWBS(15.3%)，但高于FACT-8D（5%）。EQ-HWB- s指标值与EQ-5D-5L指标值强相关（r=0.73）， FACT-8D指标值强相关（r=0.70）， EQ-HWB水平总分与FACT-G强相关（r=0.69），与WEMWBS中度相关（r=0.49）。EQ- hwb和EQ- hwb - s在已知组中的区别与EQ- 5d - 5l和事实- 8d相比，EQ- hwb - s根据EQ VAS分组、症状数量和一般健康状况具有较大的效应量，并且表现出优异的仪器水平测试-重测信度（类内相关性=0.79-0.83）和可接受的反应性（标准化反应均值=|0.24|至|0.97|）。结论：本研究代表了EQ-HWB和EQ-HWB- s在任何临床人群中的首次验证之一。两种仪器版本都显示出强大的心理测量性能。EQ-HWB-S可以推荐为乳腺癌治疗的资源分配决策提供信息。

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引用次数: 0

A New Approach for Assessing the Value of Informal Care in Alzheimer's Disease. 评估阿尔茨海默病非正式护理价值的新方法。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-12-27 DOI: 10.1016/j.jval.2024.10.3856

Anais Cheneau, Thomas Rapp

Objectives: Given that most informal caregivers providing help for patients with Alzheimer's disease are retired spouses or unemployed people, there is no market value for their time. Most articles that tried to estimate the cost of informal care for Alzheimer's disease rely on the so-called "replacement" methodology, which assumes that 1 hour of informal care has the same value as 1 hour of professional care. Little attention has been dedicated to exploring the validity of this assumption. In this article, we determine the relationship between the price of informal caregiving and professional care from the first-order condition of a theoretical model that maximizes informal caregivers' satisfaction with providing care.

Methods: This article formalizes the marginal substitution rate between informal and formal care. We assume that the caregiver's utility depends on the caregiver's burden and the patient's quality of life (QoL). After explaining the parameters of the marginal utility of caregivers, we estimate each of these parameters using PLASA data.

Results: Our results show how the value of informal care increases as the care contributes to improving patients' QoL but decreases as the burden on the caregiver increases and professionals contribute to patients' QoL.

Conclusions: The central assumption of the replacement cost method of perfect substitution between informal and formal care leads to a misestimation of the value of informal care. The effects of informal care must be considered (direct effect on the burden and indirect effects on the patient's QoL).

目的：由于大多数为阿尔茨海默病患者提供帮助的非正式照顾者是退休配偶或失业人员，因此他们的时间没有市场价值。大多数试图估算AD非正式护理成本的文章都依赖于所谓的“替代”方法，该方法假设一小时的非正式护理与一小时的专业护理具有相同的价值。很少有人关注这一假设的有效性。本文从非正式照护者提供照护满意度最大化的理论模型的一阶条件出发，确定了非正式照护价格与专业照护价格之间的关系。方法：对非正规护理和正规护理的边际替代率进行形式化分析。我们假设照顾者的效用取决于照顾者的负担和病人的生活质量。在解释了照顾者边际效用的参数后，我们使用PLASA数据估计了每个参数。结果：我们的研究结果表明，非正式护理的价值随着护理对改善患者生活质量的贡献而增加，但随着护理者负担的增加和专业人员对患者生活质量的贡献而降低。结论：非正规护理与正规护理完全替代的重置成本法的中心假设导致了对非正规护理价值的错误估计。必须考虑非正式护理的影响（对负担的直接影响和对患者生活质量的间接影响）。

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